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Gene Therapy Net is the web resource for patients and professionals interested in gene therapy. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas. In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international regulations and guidelines associated with clinical gene therapy trials.


 
Posted on: 21 August 2014, source: Sci-News.com
Dr Juliana Small of the University of Pennsylvania, Drs Raj Kurupati, Xianqyang Zhou and their colleagues from the Wistar Institute have developed a novel adenoviral vector for delivery of multiple transgenes.

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Posted on: 6 August 2014, source: The Independent
Scientists have performed a “seamless” correction to a faulty gene behind an inherited form of anaemia using a revolutionary new technique in genome editing that could transform the treatment of many genetic diseases. Two mutations in the haemoglobin gene of a patient with beta thalassemia – which can cause severe anaemia – were corrected without any errors using the Crispr technique of genome editing, the researchers said. The experiment involved converting the patient’s skin cells into stem cells in the laboratory so that the faulty gene could be corrected before the stem cells were allowed to mature into red blood cells. Without the genome correction, the red cells would have become deformed and sickle-shaped as a result of the defective haemoglobin gene.

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Posted on: 1 August 2014, source: WNCN
A new technology that allows genes to be injected into hearts with damaged electrical systems may replace the need for pacemaker implants in humans in the future. In the United States alone, there are more than 500,000 patients that get pacemaker implants annually. When the batteries on the Pacemakers run out in seven to 10 years, another surgery is required to implant a new device.

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Posted on: 1 July 2014, source: European Research Media Center
New virus serotypes are safely used as ‘DNA transporters’ to successfully deliver genes to deficient cells. Between 30 and 40 million people in Europe suffer from rare diseases—many of them children. As most of these diseases have genetic origins, gene therapy is a major hope for their future cure. Until now, however, there have been very few successful trials. Now, the EU-funded project AIPGENE, due to be completed in 2014, may have made significant progress in a gene therapy approach.
The project focussed on the genetic liver disorder, Acute Intermittent Porphyria (AIP). Through an early stage clinical trial, in phase I, it demonstrated the viability of a new approach, based on a so-called, adeno-associated vector (AAV). This is a ‘DNA transporter’ derived from a type of virus and carries the therapeutic gene to liver cells, known as hepatocytes.

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Posted on: 26 May 2014, source: NIH
In a milestone for the field of gene therapy, the National Institutes of Health (NIH) will no longer subject all proposed gene therapy clinical trials to review by a special federal advisory committee. “Given the progress in the field, I am confident that the existing regulatory authorities can effectively review most gene transfer protocols and that a streamlined process will reduce duplication and delays in getting gene transfer trials initiated,” said NIH Director Francis Collins in a statement today. Instead, the 40-year-old Recombinant DNA Advisory Committee (RAC) will review only a few trials that pose special risks.

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Posted on: 14 May 2014, source: MetroNews
An injection that promises to end obesity seems like the type of claim found only on obnoxious flashing web ads, but it’s entirely plausible that one day we will be able to treat this common problem with just the prick of a needle, according to Jason Dyck, a researcher at the University of Alberta. Two years ago, Dyck and his colleagues published a paper in the journal Nutrition and Diabetes that concluded an injectable adiponectin gene therapy reduced fat and improved insulin sensitivity in mice, despite the fact the test animals were being fed a high-fat diet.

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